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About PDGFRA D842

PDGFRA D842 is a specific mutation within the platelet-derived growth factor receptor alpha (PDGFRA), a receptor tyrosine kinase crucial for cell growth and survival. The D842 mutation leads to constitutive receptor activation, driving uncontrolled cell proliferation and contributing to disease development.

1
Approved Drugs
1
Companies
6
Indications
1
Therapeutic Areas
Broadest Approval
AYVAKIT
BLUEPRINT MEDICINES
6
approved indications

Top PDGFRA D842 Drugs

AYVAKIT
BLUEPRINT MEDICINES
6 indications · 2020
🏢

BLUEPRINT MEDICINES is the only company with an approved drug targeting PDGFRA D842.

PDGFRA D842 Drug Modality Landscape

Modalities

Small molecule
1
100%

Routes of Administration

💊 Oral
1
100%
💡

Only one approved drug targets PDGFRA D842, using small molecule modality.

Exploring alternative modalities like antibodies or PROTACs could provide differentiation and address resistance mechanisms.

Oral option available Small molecules only

PDGFRA D842 Clinical Trials 17 trials

Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on PDGFRA D842. Source: ClinicalTrials.gov. How we count

17
Total Trials
10
Active
7
Completed
100%
Completion Rate

Completion by Phase

Phase Total Completed Stopped Active Completion
Phase 1 6 4 0 2 100%
Phase 2 8 1 0 7 100%
Phase 3 1 1 0 0 100%
Phase 4 2 1 0 1 100%

Top Sponsors

Blueprint Medicines Corporat... 9 100%
Children's Hospital of Sooch... 2
Centre Leon Berard 1
H. Lee Moffitt Cancer Center... 1
M.D. Anderson Cancer Center 1
The First Affiliated Hospita... 1
Institute of Hematology & Bl... 1
Ruijin Hospital 1

By Modality

Small molecule
17 100%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

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Deep insights for drug target analysis

Competitive Landscape

  • • 1 companies competing
  • • Market share by company

Full Drug Portfolio

  • • All 1 approved drugs
  • • Approval dates & indications

Genetic Validation

  • • Full genetic evidence table
  • • Effect sizes & directions

Approval Timeline

  • • Full 1-drug timeline
  • • First-of-modality markers

Clinical Trials Analysis

  • • Sponsors: 8 (1 company)
  • • Completion rates by condition
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Full summary • All drugs • Genetic evidence • Trials • Timeline

How We Count Trials

  • Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on PDGFRA D842. A trial counts if an approved drug acting on PDGFRA D842 is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
  • Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
  • Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
  • Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
  • Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.